Bilateral lung transplantation in cystic fibrosis with hepatitis C infection - a study of two cases.

Bilateral lung transplantation in cystic fibrosis with hepatitis C infection - a study of two cases. Kardiochir Torakochirurgia Pol. 2019 Oct;16(3):133-135 Authors: Ruszel N, Kubisa B, Lisowski P, Piotrowska M, Kubisa MJ, Brykczyński M, Wojtyś M, Pieróg J, Czarnecka M, Wójcik J, Wójcik N, Sielicki P, Bielewicz M, Grodzki T PMID: 31708987 [PubMed]
Source: Polish Journal of Cardio-Thoracic Surgery - Category: Cardiovascular & Thoracic Surgery Tags: Kardiochir Torakochirurgia Pol Source Type: research

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There ’s been an odd-sounding word circulating in the English headlines recently.The word is Orkambi; a drug created by Vertex that treats approximately 40% of people born with the genetic condition, cystic fibrosis. It is the second drug to market in a promising pipeline that targets the CFTR mutation.   True gene therapy it isn’t, but it represents a near-first for a biotech firm, by successfully altering the shape and function of a protein[1]. And priced at £105,000 per patient per year, it is the latest in a long line of medicines the NHS can’t afford.Orkambi has been licensed by the EMAsin...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news
The advent of direct-acting antivirals (DAAs) in 2014 has transformed the landscape of hepatitis C virus (HCV) treatment, allowing patients to achieve cure after 8 to 12  weeks of safe and well-tolerated therapy.1 Because of the already limited organ donor pool and the availability of high numbers of HCV-viremic donors resulting from the opioid epidemic, this development has led to immense interest in transplanting organs from HCV-viremic donors into nonviremic rec ipients.2 In 2017, the first pilot of 10 noninfected patients who received kidney transplants from HCV-viremic donors demonstrated excellent allograft and ...
Source: The Journal of Thoracic and Cardiovascular Surgery - Category: Cardiovascular & Thoracic Surgery Authors: Tags: Case Report Source Type: research
Source: The Journal of Thoracic and Cardiovascular Surgery - Category: Cardiovascular & Thoracic Surgery Authors: Source Type: research
By DAVID SHAYWITZ, MD The appeal of precision medicine is the promise that we can understand disease with greater specificity and fashion treatments that are more individualized and more effective. A core tenet (or “central dogma,” as I wrote in 2015) of precision medicine is the idea that large disease categories – like type 2 diabetes – actually consist of multiple discernable subtypes, each with its own distinct characteristics and genetic drivers. As genetic and phenotypic research advances, the argument goes, diseases like “type 2 diabetes” will go the way of quaint descriptive...
Source: The Health Care Blog - Category: Consumer Health News Authors: Tags: OP-ED Uncategorized Broad Institute CAD Circulation Disease Categories Gimish Model of Disease Kathiresan Khera Massachussetts General Hospital Source Type: blogs
By DAVID SHAYWITZ, MD The appeal of precision medicine is the promise that we can understand disease with greater specificity and fashion treatments that are more individualized and more effective. A core tenet (or “central dogma,” as I wrote in 2015) of precision medicine is the idea that large disease categories – like type 2 diabetes – actually consist of multiple discernable subtypes, each with its own distinct characteristics and genetic drivers. As genetic and phenotypic research advances, the argument goes, diseases like “type 2 diabetes” will go the way of quaint descriptive...
Source: The Health Care Blog - Category: Consumer Health News Authors: Tags: Uncategorized Broad Institute CAD Circulation Disease Categories Gimish Model of Disease Kathiresan Khera Massachussetts General Hospital Source Type: blogs
By DAVID SHAYWITZ, MD The appeal of precision medicine is the promise that we can understand disease with greater specificity and fashion treatments that are more individualized and more effective. A core tenet (or “central dogma,” as I wrote in 2015) of precision medicine is the idea that large disease categories – like type 2 diabetes – actually consist of multiple discernable subtypes, each with its own distinct characteristics and genetic drivers. As genetic and phenotypic research advances, the argument goes, diseases like “type 2 diabetes” will go the way of quaint descriptive...
Source: The Health Care Blog - Category: Consumer Health News Authors: Tags: Uncategorized Broad Institute CAD Circulation Disease Categories Gimish Model of Disease Kathiresan Khera Massachussetts General Hospital Source Type: blogs
By: Janet Woodcock, M.D. FDA helps bring precision medicine – in the form of targeted therapies — to people living with diseases that have specific genetic features. Two recent FDA drug approvals point to an encouraging future for “precision medicine” — an approach for disease treatment that tailors medical therapies, including medications, to the needs of individual patients. These approvals involve diseases resulting from particular genetic characteristics identified by laboratory testing. In mid-May, FDA announced that we expanded the approval of Kalydeco (ivacaftor), enabling a larger num...
Source: Mass Device - Category: Medical Devices Authors: Tags: Blog FDA Voice Source Type: news
This study, once again disproves the misleading claims that pharmacy benefit managers and insurers (among others) make about spending on new innovative medicines. The report found that predictions of health care costs made prior to the introduction of new medicines were often dramatically overstated. The study points to the example of new cholesterol-lowering medicines, also known as PCSK9 inhibitors. The Institute for Clinical and Economic Review (ICER) predicted that the one-year cost of the two PCSK9 inhibitors would be $7.2 billion. However, in reality, based on reported sales, the actual cost will be approximately $8...
Source: Policy and Medicine - Category: American Health Authors: Source Type: blogs
So-called specialty drugs can represent lifesaving advances in the treatment of serious complex conditions, such as cancers, hepatitis C, rheumatoid arthritis, multiple sclerosis, and inflammatory bowel disease. They can turn once routinely lethal diseases such as HIV and cystic fibrosis into manageable chronic conditions. They can address rare genetic conditions, such as hemophilia, or suppress immune rejection after organ transplants.
Source: UCSF School of Pharmacy News - Category: Universities & Medical Training Authors: Source Type: news
The trends are clear: patients and institutions across the nation are concerned about skyrocketing drug prices. This post offers some information about drug pricing, explores the notion of market intervention, and proposes a series of responses to high pharmaceutical costs. A few jaw-dropping facts quickly illustrate the pattern of rising drug costs. The average annual cost of cancer drugs increased from roughly $10,000 before 2000 to over $100,000 by 2012, according to a recent study in Mayo Clinic Proceedings. Several breakthrough specialty medications and orphan drugs recently approved by the Food and Drug Administratio...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Costs and Spending Drugs and Medical Technology Featured Medicaid and CHIP Payment Policy Public Health Big Pharma CMS FDA Gilead Sciences hepatitis C Pricewaterhouse Coopers Source Type: blogs
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